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Giant Axonal Neuropathy (GAN)

WHAT IS GAN:

Giant Axonal Neuropathy (GAN) is a recessively inherited condition that results in progressive nerve death. GAN generally appears in early childhood and progresses slowly as neuronal injury becomes more severe. As the disorder progresses, patients become quadriplegics, dependent on a feeding tube and ventilator before dying typically in the second or third decade. However, we now have some confirmed GAN cases that are milder progressing, and also have confirmed cases with straight hair. The doctors working with Hannah’s Hope Fund now suspect that CMT Type 2 patients, whose causal gene has not yet been identified, may have GAN. Hannah’s Hope Fund has a collaborator for GAN gene sequencing. For more information, visit the Doctors and Families page.

OUR MISSION:

Hannah’s Hope Fund for Giant Axonal Neuropathy, Inc. is a 501(c)(3) public charity whose mission is to raise funds to support the development of treatments and a cure for GAN, and to be the resource for doctors, scientists and families world-wide.

HOW YOU CAN HELP:

Donate Now or join the many GAN families across the Globe and help us raise funds for a treatment and cure which will save their precious lives. Together these families have all come together to help save their children, but they need your help. Help them raise funds toward the continued development of the gene therapy and a therapeutic drug approach. Help Hannah’s Hope Fund spread awareness so that other families will know they are not alone.

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Email Address:

Hannah & Dr. Steve Grey at the NIH

GAN Human Clinical Trial Update

A message from Lori Sames, Hannah’s Hope CEO and Mother of Hannah:

We are thrilled that Hannah finally received gene therapy in July. While we cannot comment on the trial, we want you to know that she is doing great, thus far!

We hope that other patients are injected soon. It is likely that GAN patients will need subsequent injections to target other cell types, or perhaps may need a higher dose, so the work of Hannah’s Hope is far from over.

GAN Human Clinical Trial Background

The GAN gene therapy clinical trial began in 2015. All pre-clinical work was 100% funded by Hannah’s Hope!

The preclinical work was performed at the University of North Carolina Gene Therapy Center. They use a genetically modified virus to deliver the GAN gene to the nerve cells of children with GAN.

This trial is the first in history to deliver gene therapy through the spinal fluid to test the potential to achieve broad treatment of the spinal cord and brain. It is a momentous step forward. – Dr. Steven Gray, UNC Lead Researcher

The GAN clinical trial is “paving the way for similar gene transfer based treatments for many other neurological disorders in which nerve cells of the spinal cord and brain need to be targeted, including spinal muscular atrophy.” – Carsten Bönnemann, MD, NIH Senior Investigator

The GAN Gene Delivery Clinical Trial is recruiting participants

HONORING CHRISSY

It is with deep sadness that we inform you that our Hero, Chrissy Grube, passed away unexpectedly on February 6, 2016 from complications following scoliosis surgery. Chrissy was just 10 years old. She was eager to volunteer in the GAN gene therapy clinical trial to help her little sister, Amanda, and other GAN children around the world.